EP4303225A3 - Polyploid adeno-associated virus vectors and methods of making and using the same - Google Patents
Polyploid adeno-associated virus vectors and methods of making and using the same Download PDFInfo
- Publication number
- EP4303225A3 EP4303225A3 EP23195775.4A EP23195775A EP4303225A3 EP 4303225 A3 EP4303225 A3 EP 4303225A3 EP 23195775 A EP23195775 A EP 23195775A EP 4303225 A3 EP4303225 A3 EP 4303225A3
- Authority
- EP
- European Patent Office
- Prior art keywords
- associated virus
- making
- methods
- same
- virus vectors
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 241000702421 Dependoparvovirus Species 0.000 title abstract 2
- 208000020584 Polyploidy Diseases 0.000 title abstract 2
- 239000013598 vector Substances 0.000 title 1
- 101710197658 Capsid protein VP1 Proteins 0.000 abstract 2
- 101800001319 Capsid protein VP3 Proteins 0.000 abstract 2
- 210000000234 capsid Anatomy 0.000 abstract 2
- 101710197665 Capsid protein VP2 Proteins 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14123—Virus like particles [VLP]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
Applications Claiming Priority (5)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762471762P | 2017-03-15 | 2017-03-15 | |
US201762520901P | 2017-06-16 | 2017-06-16 | |
US201862630558P | 2018-02-14 | 2018-02-14 | |
EP18768025.1A EP3610023B1 (en) | 2017-03-15 | 2018-03-15 | Polyploid adeno-associated virus vectors and methods of making and using the same |
PCT/US2018/022725 WO2018170310A1 (en) | 2017-03-15 | 2018-03-15 | Polyploid adeno-associated virus vectors and methods of making and using the same |
Related Parent Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EP18768025.1A Division EP3610023B1 (en) | 2017-03-15 | 2018-03-15 | Polyploid adeno-associated virus vectors and methods of making and using the same |
Publications (2)
Publication Number | Publication Date |
---|---|
EP4303225A2 EP4303225A2 (en) | 2024-01-10 |
EP4303225A3 true EP4303225A3 (en) | 2024-04-10 |
Family
ID=63522576
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EP23195775.4A Pending EP4303225A3 (en) | 2017-03-15 | 2018-03-15 | Polyploid adeno-associated virus vectors and methods of making and using the same |
EP18768025.1A Active EP3610023B1 (en) | 2017-03-15 | 2018-03-15 | Polyploid adeno-associated virus vectors and methods of making and using the same |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EP18768025.1A Active EP3610023B1 (en) | 2017-03-15 | 2018-03-15 | Polyploid adeno-associated virus vectors and methods of making and using the same |
Country Status (8)
Country | Link |
---|---|
EP (2) | EP4303225A3 (zh) |
JP (1) | JP7132934B2 (zh) |
CN (2) | CN117801075A (zh) |
AU (1) | AU2018234695A1 (zh) |
CA (1) | CA3054711A1 (zh) |
ES (1) | ES2966692T3 (zh) |
IL (1) | IL268891A (zh) |
WO (1) | WO2018170310A1 (zh) |
Families Citing this family (14)
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WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
PL3356390T3 (pl) | 2015-09-28 | 2021-07-05 | The University Of North Carolina At Chapel Hill | Sposoby i kompozycje dla wektorów wirusowych unikających przeciwciał |
US10550405B2 (en) | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
WO2019006418A2 (en) | 2017-06-30 | 2019-01-03 | Intima Bioscience, Inc. | ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY |
JP7406677B2 (ja) | 2018-04-03 | 2023-12-28 | ギンコ バイオワークス インコーポレイテッド | 抗体を回避するウイルスベクター |
AU2018422759A1 (en) * | 2018-05-07 | 2020-01-02 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
US20200069819A1 (en) * | 2018-05-14 | 2020-03-05 | Biomarin Pharmaceutical Inc. | Stable expression of aav vectors in juvenile subjects |
AU2019379176A1 (en) * | 2018-11-16 | 2021-06-03 | Spark Therapeutics, Inc. | In vitro assay for detecting enhancers and inhibitors of adeno associated virus (AAV) vector transduction and/or detecting or quantitating anti-AAV binding antibodies |
WO2020142236A1 (en) * | 2019-01-02 | 2020-07-09 | Intima Bioscience, Inc. | Modified adeno-associated viral vectors for use in genetic engineering |
AU2020257182A1 (en) * | 2019-04-15 | 2021-12-09 | Spirovant Sciences, Inc. | Methods and compositions for transgene expression |
CA3157700A1 (en) | 2019-10-17 | 2021-04-22 | Stridebio, Inc. | Adeno-associated viral vectors for treatment of niemann-pick disease type c |
CN111349148A (zh) * | 2020-03-13 | 2020-06-30 | 辉大(上海)生物科技有限公司 | 一种腺相关病毒载体及其用途 |
WO2023122669A1 (en) * | 2021-12-21 | 2023-06-29 | Research Institute At Nationwide Children's Hospital | Materials and methods for the treatment of limb girdle muscular dystrophy |
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US5916563A (en) | 1988-11-14 | 1999-06-29 | United States Of America | Parvovirus protein presenting capsids |
US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
ES2026826A6 (es) | 1991-03-26 | 1992-05-01 | Ercros Sa | Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados. |
US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
US5869248A (en) | 1994-03-07 | 1999-02-09 | Yale University | Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences |
US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
US5599706A (en) | 1994-09-23 | 1997-02-04 | Stinchcomb; Dan T. | Ribozymes targeted to apo(a) mRNA |
US6040183A (en) | 1995-06-07 | 2000-03-21 | University Of North Carloina At Chapel Hill | Helper virus-free AAV production |
US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
WO1996040272A1 (en) | 1995-06-07 | 1996-12-19 | University Of North Carolina At Chapel Hill | Aav transduction of myoblasts |
WO1997022250A1 (en) | 1995-12-15 | 1997-06-26 | Intronn Llc | Therapeutic molecules generated by trans-splicing |
US6083702A (en) | 1995-12-15 | 2000-07-04 | Intronn Holdings Llc | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
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US20090215879A1 (en) | 2008-02-26 | 2009-08-27 | University Of North Carolina At Chapel Hill | Methods and compositions for adeno-associated virus (aav) with hi loop mutations |
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US10392632B2 (en) | 2011-02-14 | 2019-08-27 | The Children's Hospital Of Philadelphia | AAV8 vector with enhanced functional activity and methods of use thereof |
US9441206B2 (en) | 2011-10-28 | 2016-09-13 | The University Of North Carolina At Chapel Hill | Cell line for production of adeno-associated virus |
PT2839014T (pt) | 2012-04-18 | 2021-03-19 | Childrens Hospital Philadelphia | ¿composição e métodos para transferência de genes altamente eficiente com a utilização de variantes de capsídeo de aav |
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KR102415896B1 (ko) * | 2015-06-23 | 2022-06-30 | 더 칠드런스 호스피탈 오브 필라델피아 | 변형된 인자 ix, 및 세포, 기관 및 조직으로 유전자를 전달하기 위한 조성물, 방법 및 용도 |
-
2018
- 2018-03-15 CA CA3054711A patent/CA3054711A1/en active Pending
- 2018-03-15 EP EP23195775.4A patent/EP4303225A3/en active Pending
- 2018-03-15 CN CN202311805114.9A patent/CN117801075A/zh active Pending
- 2018-03-15 JP JP2019545960A patent/JP7132934B2/ja active Active
- 2018-03-15 CN CN201880031964.9A patent/CN110770346B/zh active Active
- 2018-03-15 AU AU2018234695A patent/AU2018234695A1/en active Pending
- 2018-03-15 ES ES18768025T patent/ES2966692T3/es active Active
- 2018-03-15 WO PCT/US2018/022725 patent/WO2018170310A1/en unknown
- 2018-03-15 EP EP18768025.1A patent/EP3610023B1/en active Active
-
2019
- 2019-08-25 IL IL26889119A patent/IL268891A/en unknown
Non-Patent Citations (5)
Title |
---|
KOHLBRENNER ET AL: "Successful Production of Pseudotyped rAAV Vectors Using a Modified Baculovirus Expression System", MOLECULAR THERAPY, CELL PRESS, US, vol. 12, no. 6, 1 December 2005 (2005-12-01), pages 1217 - 1225, XP005176630, ISSN: 1525-0016 * |
LILI WANG ET AL: "Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids", MOLECULAR THERAPY, vol. 23, no. 12, 1 December 2015 (2015-12-01), US, pages 1877 - 1887, XP055454604, ISSN: 1525-0016, DOI: 10.1038/mt.2015.179 * |
MARIA W. A. DE BACKER: "Optimization of viral vector technology to study gene function in the hypothalamus", DOCTORAL THESIS, 3 June 2010 (2010-06-03), XP055755398, Retrieved from the Internet <URL:https://dspace.library.uu.nl/bitstream/handle/1874/44371/backer.pdf?sequence=1&isAllowed=y> [retrieved on 20201201] * |
RABINOWITZ JOSEPH E. ET AL: "Cross-Dressing the Virion: the Transcapsidation of Adeno-AssociatedVirus Serotypes Functionally Defines Subgroups", JOURNAL OF VIROLOGY, vol. 78, no. 9, 1 May 2004 (2004-05-01), US, pages 4421 - 4432, XP055755326, ISSN: 0022-538X, DOI: 10.1128/JVI.78.9.4421-4432.2004 * |
ZHENG CHAI ET AL: "Chimeric Capsid Proteins Impact Transduction Efficiency of Haploid Adeno-Associated Virus Vectors", VIRUSES, vol. 11, no. 12, 9 December 2019 (2019-12-09), pages 1138, XP055755286, DOI: 10.3390/v11121138 * |
Also Published As
Publication number | Publication date |
---|---|
CN110770346A (zh) | 2020-02-07 |
CN117801075A (zh) | 2024-04-02 |
CN110770346B (zh) | 2024-01-12 |
ES2966692T3 (es) | 2024-04-23 |
JP7132934B2 (ja) | 2022-09-07 |
AU2018234695A1 (en) | 2019-09-12 |
EP4303225A2 (en) | 2024-01-10 |
CA3054711A1 (en) | 2018-09-20 |
EP3610023B1 (en) | 2023-10-04 |
JP2020511127A (ja) | 2020-04-16 |
EP3610023A4 (en) | 2021-01-20 |
WO2018170310A1 (en) | 2018-09-20 |
EP3610023A1 (en) | 2020-02-19 |
IL268891A (en) | 2019-10-31 |
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